Introduction:
Glucocorticoids (GCs) remain the gold standard for Duchenne Muscular Dystrophy (DMD) management, but long-term use is associated with adverse events (AEs) that vary by age. This study assessed the age-stratified prevalence of observed adverse clinical outcomes across neurobehavioral, endocrine/metabolic, gastrointestinal, musculoskeletal, infectious and ophthalmologic categories among Medicaid patients on long-term GC therapy.
Methods:
A retrospective analysis using 2016-2022 Medicaid T-MSIS Files identified male DMD patients aged ≤30 years with continuous enrollment. Long-term GC use was defined as ≥90 days with ≤60-day gaps between refills. Clinically relevant age stratifications examined observed adverse clinical outcomes using ICD-10 diagnosis codes.
Results:
Among 1,365 patients (mean age: 12 years; 39% White, 27% Hispanic), most resided in California (17%), Texas (10%), and New York (9%). Mean GC duration was 2 years, with 22% on Deflazacort only, 45% on Prednisone only, and 32% using either medication at separate times. Age-stratified analyses revealed significant differences in neurobehavioral changes, musculoskeletal conditions (fractures, osteoporosis, scoliosis), endocrine/metabolic AEs (delayed puberty, impaired growth, obesity) and infections (p<0.05). Insomnia/sleep disorders were the most common neurobehavioral conditions, affecting 67% of patients. Anxiety, dissociative and somatoform disorders affected 22% overall, peaking at 27% at 14-19 years. Upper respiratory infections were the most frequent AEs (55%), and highest at 77% among ages 4–7. Pneumonia (17%), septicemia (11%) and UTIs (8%) all peaked at 20–30 years. Bone fractures (long bones and vertebrae) were most prevalent in the 8–13 age group (35%), with obesity also peaking within the same age group at 42%. Cataracts affected 10% of patients but did not vary by age.
Conclusion:
Observed adverse clinical outcomes including neurobehavioral, metabolic, musculoskeletal and infectious complications, were highly prevalent and demonstrated significant age-related variations. These findings highlight the critical need for DMD therapies that reduce AEs while maintaining efficacy and slowing disease progression.